Melphalan, lenalidomide and dexamethasone for the treatment of immunoglobulin light chain amyloidosis: results of a phase II trial.

Abstract:

:We report results of a phase II trial of combination of melphalan, lenalidomide, and dexamethasone for the treatment of immunoglobulin light chain (AL) amyloidosis. The primary objectives were tolerability and hematologic response rate; secondary objectives were organ responses and survival. Treatment protocol consisted of melphalan 5 mg/m(2)/day for four days, lenalidomide 10 mg/day for 21 days and dexamethasone 20-40 mg once a week every 28 days for a total of 12 cycles. Sixteen subjects were enrolled of whom 14 completed at least 3 cycles and were evaluable for response. Grade 3/4 toxicities were experienced by 88% (n=14), the most common being myelosuppression (n=7). Dose reductions occurred in 85% (n=12 of 14) of subjects. Hematologic partial and complete responses were achieved by 43% (n=6 of 14) and 7% (n=1 of 14), respectively. The median overall survival has not been reached and median progression-free survival is 24 months. In conclusion, this combination is associated with significant myelosuppression leading to dose modifications and producing minor hematologic responses in AL amyloidosis. http://clinicaltrials.gov/ct2/show/NCT00679367.

journal_name

Haematologica

journal_title

Haematologica

authors

Sanchorawala V,Patel JM,Sloan JM,Shelton AC,Zeldis JB,Seldin DC

doi

10.3324/haematol.2012.075192

subject

Has Abstract

pub_date

2013-05-01 00:00:00

pages

789-92

issue

5

eissn

0390-6078

issn

1592-8721

pii

haematol.2012.075192

journal_volume

98

pub_type

杂志文章
  • Identification of a novel fusion, SQSTM1-ALK, in ALK-positive large B-cell lymphoma.

    abstract::ALK-positive large B-cell lymphoma is a rare subtype of lymphoma, and most cases follow an aggressive clinical course with a poor prognosis. We examined an ALK-positive large B-cell lymphoma case showing an anti-ALK immunohistochemistry pattern distinct from those of 2 known ALK fusions, CLTC-ALK and NPM-ALK, for the ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2010.033514

    authors: Takeuchi K,Soda M,Togashi Y,Ota Y,Sekiguchi Y,Hatano S,Asaka R,Noguchi M,Mano H

    更新日期:2011-03-01 00:00:00

  • Long-term follow up of the FL2000 study comparing CHVP-interferon to CHVP-interferon plus rituximab in follicular lymphoma.

    abstract::Anti-CD20-containing chemotherapy regimens have become the standard of care for patients with follicular lymphoma needing cytotoxic therapy. Four randomized trials demonstrated a clinical benefit for patients treated with rituximab. However, no long-term follow up (i.e. > 5 years) of these trials is yet available. Bet...

    journal_title:Haematologica

    pub_type: 杂志文章,随机对照试验

    doi:10.3324/haematol.2012.082412

    authors: Bachy E,Houot R,Morschhauser F,Sonet A,Brice P,Belhadj K,Cartron G,Audhuy B,Fermé C,Feugier P,Sebban C,Delwail V,Maisonneuve H,Le Gouill S,Lefort S,Brousse N,Foussard C,Salles G,Groupe d'Etude des Lymphomes de l'Adult

    更新日期:2013-07-01 00:00:00

  • Multidrug resistant Pseudomonas aeruginosa infection in children undergoing chemotherapy and hematopoietic stem cell transplantation.

    abstract::Pseudomonas aeruginosa is one leading gram-negative organism associated with nosocomial infections. Bacteremia is life-threatening in the immunocompromised host. Increasing frequency of multi-drug-resistant (MDRPA) strains is concerning. We started a retrospective survey in the pediatric hematology oncology Italian ne...

    journal_title:Haematologica

    pub_type: 杂志文章,多中心研究

    doi:10.3324/haematol.2009.020867

    authors: Caselli D,Cesaro S,Ziino O,Zanazzo G,Manicone R,Livadiotti S,Cellini M,Frenos S,Milano GM,Cappelli B,Licciardello M,Beretta C,Aricò M,Castagnola E,Infection Study Group of the Associazione Italiana Ematologia Oncologia Pediat

    更新日期:2010-09-01 00:00:00

  • Familial lymphoid neoplasms in patients with mantle cell lymphoma.

    abstract:BACKGROUND AND OBJECTIVES:Familial aggregation has been recognized in patients with several lymphoid neoplasms, but the genetic basis for this familial clustering is not known. Germ-line mutations in the ataxia-telangiectasia mutated (ATM) and CHK2 genes have been detected in patients with mantle cell lymphoma (MCL), s...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Tort F,Camacho E,Bosch F,Harris NL,Montserrat E,Campo E

    更新日期:2004-03-01 00:00:00

  • Neurological disorders in essential thrombocythemia.

    abstract::Patients with essential thrombocythemia often complain of various subjective neurological symptoms. This prospective study aims to assess their incidence and response to therapy. Among 37 consecutive patients with essential thrombocythemia, 11 presented with neurological symptoms. Among them 4 had thrombotic events, 7...

    journal_title:Haematologica

    pub_type: 临床试验,杂志文章

    doi:10.3324/haematol.2011.050005

    authors: Billot S,Kouroupi EG,Le Guilloux J,Cassinat B,Jardin C,Laperche T,Fenaux P,Carpentier AF,Kiladjian JJ

    更新日期:2011-12-01 00:00:00

  • Respective prognostic values of germinal center phenotype and early (18)fluorodeoxyglucose-positron emission tomography scanning in previously untreated patients with diffuse large B-cell lymphoma.

    abstract:BACKGROUND AND OBJECTIVES:Diffuse large B-cell lymphomas (DLBCL) have a variable outcome, and powerful methods of prognostication are needed in order to choose the best treatment for each patient. Immunophenotypic classification of the tumor as germinal center (GC) or non-germinal center-like (nGC) and early response e...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.10895

    authors: Dupuis J,Gaulard P,Hemery F,Itti E,Gisselbrecht C,Rahmouni A,Copie-Bergman C,Brière J,El Gnaoui T,Gaillard I,Meignan M,Haioun C

    更新日期:2007-06-01 00:00:00

  • Expression of the c-Met oncogene by tumor cells predicts a favorable outcome in classical Hodgkin's lymphoma.

    abstract:BACKGROUND:The c-Met signaling pathway regulates a variety of biological processes, including proliferation, survival and migration. Deregulated c-Met activation has been implicated in the pathogenesis and prognosis of many human malignancies. We studied the function and prognostic significance of c-Met and hepatocyte ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2011.056101

    authors: Xu C,Plattel W,van den Berg A,Rüther N,Huang X,Wang M,de Jong D,Vos H,van Imhoff G,Viardot A,Möller P,Poppema S,Diepstra A,Visser L

    更新日期:2012-04-01 00:00:00

  • Fludarabine-based chemotherapy in untreated mantle cell lymphomas: an encouraging experience in 29 patients.

    abstract:BACKGROUND AND OBJECTIVE:A prospective study to evaluate the role of fludarabine alone or in combination with idarubicin in untreated patients with mantle cell lymphoma (MCL). DESIGN AND METHODS:Twenty-nine untreated patients with mantle cell lymphoma were stochastically treated with intravenous fludarabine at a dose ...

    journal_title:Haematologica

    pub_type: 临床试验,杂志文章

    doi:

    authors: Zinzani PL,Magagnoli M,Moretti L,Battista R,Ronconi F,De Renzo A,Zaccaria A,Gentilini P,Guardigni L,Gherlinzoni F,Cellini C,Fattori PP,Bendandi M,Bocchia M,Aitini E,Tura S

    更新日期:1999-11-01 00:00:00

  • Heterogeneous breakpoints in patients with acute lymphoblastic leukemia and the dic(9;20)(p11-13;q11) show recurrent involvement of genes at 20q11.21.

    abstract::The dic(9;20)(p11-13;q11) is a recurrent chromosomal abnormality in patients with acute lymphoblastic leukemia. Although it results in loss of material from 9p and 20q, the molecular targets on both chromosomes have not been fully elucidated. From an initial cohort of 58 with acute lymphoblastic leukemia patients with...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2008.002808

    authors: An Q,Wright SL,Moorman AV,Parker H,Griffiths M,Ross FM,Davies T,Harrison CJ,Strefford JC

    更新日期:2009-08-01 00:00:00

  • Risk factors for the development of bacterial infections in multiple myeloma treated with two different vincristine-adriamycin-dexamethasone schedules.

    abstract:BACKGROUND AND OBJECTIVES:We evaluated bacterial infections (BIs) in patients with multiple myeloma (MM) treated with two different schedules of vincristine-adriamycin-dexamethasone (VAD). DESIGN AND METHODS:Ninety-seven patients were studied during 340 VAD cycles. VAD was given by either continuous intravenous infusi...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Cesana C,Nosari AM,Klersy C,Miqueleiz S,Rossi V,Ferrando P,Valentini M,Barbarano L,Morra E

    更新日期:2003-09-01 00:00:00

  • Difference in gene expression between human fetal liver and adult bone marrow mesenchymal stem cells.

    abstract:BACKGROUND AND OBJECTIVES:Mesenchymal stem cells (MSC) are progenitor cells that are capable of differentiating into mesenchymal tissues. Fetal and adult MSC have similar morphology but differ in proliferative, differentiating and immunosuppressive properties. Further exploring their differences could help in choosing ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Götherström C,West A,Liden J,Uzunel M,Lahesmaa R,Le Blanc K

    更新日期:2005-08-01 00:00:00

  • Krüppel-like factor 4 blocks tumor cell proliferation and promotes drug resistance in multiple myeloma.

    abstract::Krüppel-like factor 4 is a transcription factor with anti-proliferative effects in differentiated cells, but with the ability to reprogram adult cells into cell-cycling pluripotent cells. In cancer, Krüppel-like factor 4 acts as either an anti-oncogene or an oncogene. We analyzed Krüppel-like factor 4 gene expression ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2012.066944

    authors: Schoenhals M,Kassambara A,Veyrune JL,Moreaux J,Goldschmidt H,Hose D,Klein B

    更新日期:2013-09-01 00:00:00

  • Kasabach-Merritt syndrome associated with giant liver hemangioma: the effect of combined therapy with danaparoid sodium and tranexamic acid.

    abstract::n patients with Kasabach-Merritt syndrome (KMS), local activation of coagulation commonly results in disseminated intravascular coagulation (DIC). Progress of DIC is associated with 30-40% mortality as a result of uncontrollable hemorrhage. A 39-year-old woman with an enlarging giant liver hemangioma was diagnosed as ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Ontachi Y,Asakura H,Omote M,Yoshida T,Matsui O,Nakao S

    更新日期:2005-11-01 00:00:00

  • The Malmö International Brother Study (MIBS). Genetic defects and inhibitor development in siblings with severe hemophilia A.

    abstract:BACKGROUND AND OBJECTIVES:The strongest risk factor identified for inhibitor development in people with severe hemophilia A is the type of factor VIII gene mutation. The objective of this study was to evaluate the mutation type dependent concordance rate of inhibitor formation in siblings. DESIGN AND METHODS:The gene ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Astermark J,Oldenburg J,Escobar M,White GC 2nd,Berntorp E,Malmö International Brother Study study group.

    更新日期:2005-07-01 00:00:00

  • Diagnostic criteria for hematopoietic stem cell transplant-associated microangiopathy: results of a consensus process by an International Working Group.

    abstract:BACKGROUND AND OBJECTIVES:There are no widely accepted criteria for the definition of hematopoietic stem cell transplant -associated microangiopathy (TAM). An International Working Group was formed to develop a consensus formulation of criteria for diagnosing clinically significant TAM. DESIGN AND METHODS:The particip...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.10699

    authors: Ruutu T,Barosi G,Benjamin RJ,Clark RE,George JN,Gratwohl A,Holler E,Iacobelli M,Kentouche K,Lämmle B,Moake JL,Richardson P,Socié G,Zeigler Z,Niederwieser D,Barbui T,European Group for Blood and Marrow Transplantation.,E

    更新日期:2007-01-01 00:00:00

  • Front-line treatment of Philadelphia positive chronic myeloid leukemia with imatinib and interferon-alpha: 5-year outcome.

    abstract::In 2004, we reported the short-term results of a multicentric, phase 2 study of imatinib 400 mg daily and pegylated interferon-alpha in the treatment of 76 early chronic phase Philadelphia-positive chronic myeloid leukemia patients. In this report, we update the results with an observation time of five years. After tw...

    journal_title:Haematologica

    pub_type: 杂志文章,多中心研究

    doi:10.3324/haematol.12265

    authors: Palandri F,Iacobucci I,Castagnetti F,Testoni N,Poerio A,Amabile M,Breccia M,Intermesoli T,Iuliano F,Rege-Cambrin G,Tiribelli M,Miglino M,Pane F,Saglio G,Martinelli G,Rosti G,Baccarani M,GIMEMA Working Party on CML.

    更新日期:2008-05-01 00:00:00

  • Estimation of bone marrow cellularity by means of vertebral magnetic resonance.

    abstract:BACKGROUND AND OBJECTIVE:A magnetic resonance (MR) signal shows an inverse correlation with bone marrow cellularity. In this study, we investigated the possibility of estimating the degree of bone marrow cellularity by means of this non-invasive technique. METHODS:In 25 patients with different hematological disorders ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Rozman M,Mercader JM,Aguilar JL,Montserrat E,Rozman C

    更新日期:1997-03-01 00:00:00

  • Detection of heterozygous large deletions in the antithrombin gene using multiplex polymerase chain reaction and denatured high performance liquid chromatography.

    abstract::The present study reports a method for the easy, rapid and cost effective detection of heterozygous large deletions. As a model gene all exons of the antithrombin gene were amplified in a one tube multiplex polymerase chain reaction (PCR) and the products separated according to their size by reverse-phase ion-pair hig...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Pavlova A,El-Maarri O,Luxembourg B,Lindhoff-Last E,Kochhan L,Bruhn HD,Delev D,Watzka M,Seifried E,Oldenburg J

    更新日期:2006-09-01 00:00:00

  • Chronic phase chronic myeloid leukemia patients with low OCT-1 activity randomized to high-dose imatinib achieve better responses and have lower failure rates than those randomized to standard-dose imatinib.

    abstract:BACKGROUND:The functional activity of the organic cation transporter 1 (OCT-1) protein (OCT-1 activity) is an excellent predictor of molecular response and progression-free survival in patients with newly diagnosed chronic phase chronic myeloid leukemia treated with imatinib as front-line therapy. DESIGN AND METHODS:I...

    journal_title:Haematologica

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.3324/haematol.2011.056457

    authors: White DL,Radich J,Soverini S,Saunders VA,Frede AK,Dang P,Cilloni D,Lin P,Mongay L,Woodman R,Manley P,Slader C,Kim DW,Pane F,Martinelli G,Saglio G,Hughes TP

    更新日期:2012-06-01 00:00:00

  • IGHV mutational status and outcome for patients with chronic lymphocytic leukemia upon treatment: a Danish nationwide population-based study.

    abstract::Patients with chronic lymphocytic leukemia and unmutated immunoglobulin heavy-chain variable region gene (IGHV) have inferior survival from time of treatment in clinical studies. We assessed real-world outcomes based on mutational status and treatment regimen in a nationwide population-based cohort, comprising all 4,1...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2019.220194

    authors: Rotbain EC,Frederiksen H,Hjalgrim H,Rostgaard K,Egholm GJ,Zahedi B,Poulsen CB,Enggard L,da Cunha-Bang C,Niemann CU

    更新日期:2020-06-01 00:00:00

  • Association of anemia with health-related quality of life and survival: a large population-based cohort study.

    abstract::Anemia is highly prevalent, especially in older individuals. In selected populations, anemia has been reported to be associated with impaired survival and health-related quality of life. However, data on this impact in the general population are rare. Furthermore, discussions on the optimal definition of anemia have n...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2018.195552

    authors: Wouters HJCM,van der Klauw MM,de Witte T,Stauder R,Swinkels DW,Wolffenbuttel BHR,Huls G

    更新日期:2019-03-01 00:00:00

  • Intensive polychemotherapy experience in chronic myeloid leukaemia.

    abstract::A pilot study, testing two different chemotherapeutic schedules in CML patients, was carried out in order to verify the possibility of Ph1 clone disappearance. Both therapeutic regimens, though able to induce severe marrow aplasia, determined only a reduction of Ph1 positive metaphases in two patients, while in one ca...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Centurioni R,Leoni P,Russo D,Brianzoni MF,Montillo M,Baccarani M,Tura S

    更新日期:1989-09-01 00:00:00

  • Verification and comparison of two different predictive equations in Hodgkin's disease.

    abstract:BACKGROUND AND OBJECTIVE:In recent years, two predictive equations to estimate median expected survival at diagnosis for patients affected with Hodgkin's disease have been developed at the University of Pavia Medical School. The present retrospective work was aimed at testing correlation between mean survival estimated...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Bettini R,Tonolini M,Maccianti E

    更新日期:1997-05-01 00:00:00

  • MicroRNA-125b transforms myeloid cell lines by repressing multiple mRNA.

    abstract:BACKGROUND:We previously described a t(2;11)(p21;q23) chromosomal translocation found in patients with myelodysplasia or acute myeloid leukemia that leads to over-expression of the microRNA miR-125b, and we showed that transplantation of mice with murine stem/progenitor cells overexpressing miR-125b is able to induce l...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2011.061515

    authors: Bousquet M,Nguyen D,Chen C,Shields L,Lodish HF

    更新日期:2012-11-01 00:00:00

  • Clinical profile and treatment outcome of older (>75 years) patients with systemic AL amyloidosis.

    abstract::Systemic AL amyloidosis, a disease with improving outcomes using novel therapies, is increasingly recognized in the elderly but treatment and outcomes have not been systematically studied in this group of patients in whom comorbidities and frailty may compound morbidity and mortality. We report the outcomes of 295 pat...

    journal_title:Haematologica

    pub_type: 临床试验,杂志文章

    doi:10.3324/haematol.2015.128025

    authors: Sachchithanantham S,Offer M,Venner C,Mahmood SA,Foard D,Rannigan L,Lane T,Gillmore JD,Lachmann HJ,Hawkins PN,Wechalekar AD

    更新日期:2015-11-01 00:00:00

  • Transition from fresh frozen plasma to solvent/detergent plasma in the Netherlands: comparing clinical use and transfusion reaction risks.

    abstract::Plasma transfusion is indicated for replenishment of coagulative proteins to stop or prevent bleeding. In 2014, the Netherlands switched from using ~300mL fresh frozen plasma (FFP) units to using 200mL Omniplasma, a solvent/detergent treated pooled plasma (SD plasma), units. We evaluated the effect of the introduction...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2019.222083

    authors: Saadah NH,Schipperus MR,Wiersum-Osselton JC,van Kraaij MG,Caram-Deelder C,Beckers EAM,Leyte A,Rondeel JMM,de Vooght KMK,Weerkamp F,Zwaginga JJ,van der Bom JG

    更新日期:2020-04-01 00:00:00

  • Dietary intake of heme iron is associated with ferritin and hemoglobin levels in Dutch blood donors: results from Donor InSight.

    abstract::Whole blood donors, especially frequently donating donors, have a risk of iron deficiency and low hemoglobin levels, which may affect their health and eligibility to donate. Lifestyle behaviors, such as dietary iron intake and physical activity, may influence iron stores and thereby hemoglobin levels. We aimed to inve...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2019.229450

    authors: Timmer TC,de Groot R,Rijnhart JJM,Lakerveld J,Brug J,Perenboom CWM,Baart MA,Prinsze FJ,Zalpuri S,van der Schoot EC,de Kort WLAM,van den Hurk K

    更新日期:2020-10-01 00:00:00

  • Cutaneous T-cell lymphomas (including rare subtypes). Current concepts. II.

    abstract:BACKGROUND:Cutaneous T-cell lymphomas (CTCL) represent about the 80% of skin lymphomas and comprise a heterogeneous group of diseases with respect to clinical presentation, outcome, histologic and immunophenotypic features. In the recent years, data have been accumulated indicating that clinical and biological differen...

    journal_title:Haematologica

    pub_type: 杂志文章,评审

    doi:

    authors: Paulli M,Berti E

    更新日期:2004-11-01 00:00:00

  • MOHITO, a novel mouse cytokine-dependent T-cell line, enables studies of oncogenic signaling in the T-cell context.

    abstract::The mouse pro-B cell line Ba/F3 has gained major interest as a model system to investigate oncogenic tyrosine kinases and to determine the efficacy of kinase inhibitors. While Ba/F3 cells are suitable to study oncogenic kinases derived from various cell types, the signaling networks in Ba/F3 cells are B-cell specific....

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2010.035931

    authors: Kleppe M,Mentens N,Tousseyn T,Wlodarska I,Cools J

    更新日期:2011-05-01 00:00:00

  • Stat5 is critical for the development and maintenance of myeloproliferative neoplasm initiated by Nf1 deficiency.

    abstract::Juvenile myelomonocytic leukemia is a rare myeloproliferative neoplasm characterized by hyperactive RAS signaling. Neurofibromin1 (encoded by the NF1 gene) is a negative regulator of RAS activation. Patients with neurofibromatosis type 1 harbor loss-of-function mutations in NF1 and have a 200- to 500-fold increased ri...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2015.136002

    authors: Sachs Z,Been RA,DeCoursin KJ,Nguyen HT,Mohd Hassan NA,Noble-Orcutt KE,Eckfeldt CE,Pomeroy EJ,Diaz-Flores E,Geurts JL,Diers MD,Hasz DE,Morgan KJ,MacMillan ML,Shannon KM,Largaespada DA,Wiesner SM

    更新日期:2016-10-01 00:00:00